The National Organization for Rare Disorders and the University of Maryland will develop a training program to help caregivers and patients participate in research.
Amgen's PCSK9 inhibitor is the second in its class to get an FDA panel endorsement; a new drug could threaten Shire's hold on a rare-disease category; a study shows pharma invests less in Hispanic audiences than in other groups
Nexium tops the list of Medicare's spending on brand drugs; Gilead Sciences doubles its first-quarter profit; Pfizer reportedly wants to buy a rare-disease drug company
The cancer and immunology drug may help patients with a condition that affects two people in every one million.
Horizon Pharma's purchase of Hyperion Therapeutics will add two rare-disease drugs to its portfolio.
The duo hope to begin testing a new treatment within three years.
The $38-million deal expands the reach of rare-disease campaigns.
Industry rare disease awareness efforts included a pipeline overview at Pfizer as well as BI's efforts to draw attention to IPF.
The FDA approved NPS Pharmaceuticals's rare-disease drug Friday. The company, which Shire shelled out $5.2 billion to buy, expects it to hit the market during the second-quarter.
The specialty biopharma firm says it's hiring new commercial managers after a buying spree.
BioMarin to acquire Dutch biotech Prosensa; Woodcock says significant efficacy key to Breakthrough Therapy designation; Stryker considers $16 billion bid for UK devicemaker Smith and Nephew.
Success today is predicated on recognizing that every patient is different, every disease is different, every drug, every breakthrough and every cure is different.
The $310,000 price for the Gaucher disease medication is typical of the rare-disease category.
FDA inspectors have cited the manufacturer of the rare-disease drug Soliris (eculizumab) for poor manufacturing practices.
The oral medication Cerdelga joins Cerezyme in its Gaucher disease arsenal. Sanofi expects the drug will be priced "on par" with Cerezyme, which goes for around $300,000 a year in the US.
Phase-III tests indicate patients may be able to switch from injectable enzyme-replacement therapies, like Fabrazyme, to the firm's oral drug.
A $225-million deal with US biotech ArmaGen gives Shire worldwide commercialization rights to an experimental enzyme replacement therapy.
An advocacy group alleges that Shire's decision to ignore the US Fabry disease market was the result of a backroom deal with Sanofi.
Analyst reaction to this week's quarterly news reflected a need to woo investors, adding to some negative carryover from the previous year.
The company said it plans to file its rare-disease drug with the FDA later this year, for a 2015 review.
The Thousand Oaks, Calif. company says its cholesterol-lowering antibody hit its clinical trial endpoint among rare disease patients.
The UK watchdog wants to know what's behind the rare-disease drug's high price.
Kynamro's slow start, coupled with competition, has prompted Sanofi to add sales reps.
Regulatory Focus discusses how the expanding US population may make it harder for a condition to qualify as rare.
The FDA granted priority review for the experimental, all-oral Gaucher's disease treatment.
The agency extended the review time for Biogen's Alprolix by three months.
The patient population is small, at around 3,000, but the expected price tag is $330,000 a year.
The catch is the difference between sales and revenues. Generics will continue to be a force, and small-audience drugs will become increasingly important.
The US-only recall amounts to less than 1% of the company's inventory.
The regulator says the drugmaker needs to do more testing.
Specialty medicines continue to be a hot category, one in which spending has surged behind products that treat medicine's most complex diseases. This eBook lays out the needs and hurdles along the specialty drug pathway, explaining how stakeholders are working to get meds into patients' hands and ease their experience. Click here to download.